Autosomal recessive cerebellar ataxia
MONDO:0015244Autosomal recessive cerebellar ataxias (ARCA) are a heterogeneous group of rare neurological disorders involving both the central and peripheral nervous system (and in some cases other systems and organs), and characterized by degeneration or abnormal development of the cerebellum and spinal cord and, in most cases, early onset occurring before the age of 20 years.
Also known as: ARCA, arca, cerebellar ataxia, autosomal recessive
90 clinical trials for this condition and its sub-types.
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Virus therapy fails to advance in colorectal cancer trial
Disease control TerminatedThis study tested a new approach using a virus that attacks cancer cells (oncolytic immunotherapy) along with two standard drugs (atezolizumab and bevacizumab) in people with advanced colorectal cancer that had stopped responding to other treatments. The trial was stopped early a…
Phase: PHASE2 • Sponsor: Replimune, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:01 UTC
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Experimental Friedreich's ataxia drug tested in kids – but trial halted early
Disease control TerminatedThis early-stage trial tested a drug called nomlabofusp (CTI-1601) in 18 adolescents and children with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to check safety and how the body processes the drug. However, the study was term…
Phase: PHASE1 • Sponsor: Larimar Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Experimental cell therapy targets deadly childhood brain cancer
Disease control TerminatedThis early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…
Phase: PHASE1 • Sponsor: University of Florida • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC